Skip to main content
Log in

Determinants of Drug Costs in Hospitalised Patients with Haemophilia

Impact of Recombinant Activated Factor VII

  • Original Research Article
  • Published:
PharmacoEconomics Aims and scope Submit manuscript

Abstract

Objective: To analyse the determinants of anti-haemophilic drug costs in hospitalised patients with haemophilia and to estimate the impact of recombinant activated factor VII (rFVIIa) therapy on this expenditure.

Perspective: The perspective of the study was from the viewpoint of the hospital.

Design and setting: A prospective study was carried out. All patients with haemophilia who were hospitalised in 1999 in Bicêtre public hospital, Paris, France were included in the cohort.

Main outcome measures and results: For each of the 96 patients (154 hospital stays), we estimated the costs of anti-haemophilic drugs (coagulation concentrates) used. Costs were then stratified by different variables (severity of the disease, presence of a circulating inhibitor to coagulation factors, etc.) and a multivariate model was developed to determine the relationship between these variables and total anti-haemophilic drug costs, while controlling for potential confounders. Our study revealed: (i) the independent role of the five following variables in contributing to high anti-haemophilic drug expenditure: presence of a circulating inhibitor to coagulation factors, odds ratio (OR) = 16.9 (95% CI: 4.3–66); severity of the disease (factor VIII or factor IX≤0.01 IU/mL), OR = 3.7 (95% CI: 1.6–8.6); length of hospital stay >4 days, OR = 8 (95% CI: 2.2–29.4); age >18 years old, OR = 6.2 (95% CI: 1.6–24.5); and surgical reasons for hospitalisation (whether surgery was haemophilia related [OR = 35.7 (95% CI: 7.3–175)] or not [OR = 5.4 (95% CI: 1.3–22.5)]); (ii) the large share that rFVIIa represented in this expenditure on medicines: rFVIIa was used in 20.1% of hospital stays and accounted for 56.2% of the total anti-haemophilic drug costs, which were estimated at €4 384 732 (2000 values).

Conclusions: Our data underline the heavy cost of the treatment of haemophilic patients with an inhibitor to coagulation factors. But, to the question of whether the high expenditure linked to rFVIIa utilisation will be balanced out by later benefits, it is not yet possible to reply with any certainty; further cost-benefit evaluation should be carried out.

This is a preview of subscription content, log in via an institution to check access.

Access this article

Price excludes VAT (USA)
Tax calculation will be finalised during checkout.

Instant access to the full article PDF.

Table I
Table II
Table III

Similar content being viewed by others

Notes

  1. Use of tradenames is for product identification only and does not imply endorsement.

References

  1. Tuddenham EGD, Cooper DN. The molecular genetics of haemostasis and its inherited disorders. Oxford monographs in medical genetics n°25. Oxford, England: Oxford University Press, 1994

    Google Scholar 

  2. Mannucci PM, Tuddenham EGD. The haemophilias — from royal genes to gene therapy. N Engl J Med 2001; 344 (23): 1773–9

    Article  PubMed  CAS  Google Scholar 

  3. Rosendaal FR, Varekamp I, Smith C, et al. Mortality and causes of death in Dutch hemophiliacs, 1973–1986. Br J Haematol 1989; 71: 71–6

    Article  PubMed  CAS  Google Scholar 

  4. Bohn RL, Avorn J, Glynn RJ, et al. Prophylactic use of factor VIII: an economic evaluation. Thromb Haemost 1998; 79 (5): 932–7

    PubMed  CAS  Google Scholar 

  5. Lee C, Sabin C, Miners A. High cost, low volume care: the case of haemophilia. BMJ 1997; 315: 962–3

    Article  PubMed  CAS  Google Scholar 

  6. Scharrer I, Bray GL, Neutzling O. Incidence of inhibitors in haemophilia A patients — a review of recent studies of recombinant and plasma-derived factor VIII concentrates. Haemophilia 1999; 5: 145–54

    Article  PubMed  CAS  Google Scholar 

  7. Novoseven®website. Available from URL: http://www.novoseven-us.com [Accessed 2003 May 15]

  8. Ross-Degna D, Soumerai SB, Avorn J, et al. Haemophilia home treatment: economic analysis and implications for health policy. Int J Technol Assess Health Care 1995; 11: 327–44

    Article  Google Scholar 

  9. Meeting report: economic aspects of haemophilia care in the United States. Haemophilia 1999; 5 (4): 282–5

    Google Scholar 

  10. Szucs TD, Offner A, Kroner B, et al. Resource utilisation in haemophiliacs treated in Europe: results from the European Study on Socioeconomic Aspects of Haemophilia Care. The European Socioeconomic Study Group. Haemophilia 1998; 4 (4): 498–501

    Article  PubMed  CAS  Google Scholar 

  11. Aldedort LM. Economic aspects of haemophilia care. Haemophilia 1999; 5 (3): 216–9

    Article  PubMed  CAS  Google Scholar 

  12. Hoyer LW. Haemophilia A. N Engl J Med 1994; 330 (1): 38–47

    Article  PubMed  CAS  Google Scholar 

  13. SAS/Stat. User’s guide: release 8.1 edition. Cary (NC): SAS Institute, 2001

    Google Scholar 

  14. Sultan Y. Prevalence of inhibitors in a population of 3435 haemophilia patients in France. French Haemophilia Study Group. Thromb Haemost 1992; 67 (6): 600–2

    PubMed  CAS  Google Scholar 

  15. Mannucci PM. Haemophilia treatment protocols around the world: towards a consensus. Haemophilia 1998; 4: 421–30

    Article  PubMed  CAS  Google Scholar 

  16. Haemophilia of Georgia. Protocols for the treatment of haemophilia and von Willebrand disease. Haemophilia 2000; 6 (1 Suppl.): 84–93

    Google Scholar 

  17. Miller DG, Stamatoyannopoulos G. Gene therapy for hemophilia. N Engl J Med 2001; 344 (23): 1782–3

    Article  PubMed  CAS  Google Scholar 

  18. Lipton RA. The economics of factor VIII inhibitor treatment. Semin Hematol 1994; 31 (2 Suppl. 4): 37–8

    PubMed  CAS  Google Scholar 

  19. Rivard GE, Vick S. Factor VIII inhibitor treatment: economics of inhibitor treatment in Canada. Semin Hematol 1994; 31 (2 Suppl. 4): 41–3

    PubMed  CAS  Google Scholar 

  20. Suczs TD, Offner A, Schramm W. Socioeconomic impact of haemophilia care: results of a pilot study. Haemophilia 1996; 2: 211–7

    Article  Google Scholar 

  21. Goudemand J. Pharmaco-economic aspects of inhibitor treatment. Eur J Haematol 1998; 61 (63 Suppl.): 24–7

    Google Scholar 

  22. Wasserman J, Ullman M, Begley C, et al. The impact of changing health care reimbursement methodologies on persons with hemophilia [abstract 191]. Haemophilia 1998; 4: 203

    Google Scholar 

  23. Chang H, Sher GD, Blanchette VS, et al. The impact of inhibitors on the cost of clotting factor replacement therapy in Haemophilia A in Canada. Haemophilia 1999; 5 (4): 247–52

    Article  PubMed  CAS  Google Scholar 

  24. Montague O, Chaix C, Harf A, et al. Costs for acute myocardial infarction in a tertiary care centre and nationwide in France. Pharmacoeconomics 2000; 17 (6): 603–9

    Article  Google Scholar 

  25. Dimichele DM. Inhibitors in Haemophilia: a primer. Haemophilia 2000; 6 (1 Suppl.): 38–40

    Article  PubMed  Google Scholar 

  26. Stewart AJ, Hanley JP, Ludlam CA. Safety, efficacy and cost-effectiveness of home therapy with recombinant activated factor VII in a patient with severe haemophilia A and an anti factor VIII inhibitor. Blood Coagul Fibrinolysis 1998; 9 (1 Suppl.): 93–5

    Google Scholar 

  27. Ingerslev J, Thykjaer H, Kudsk Jensen O, et al. Home treatment with recombinant activated factor VII: results from one centre. Blood Coagul Fibrinolysis 1998; 9 (1 Suppl.): 107–10

    Google Scholar 

  28. Ekert H, Brewin T, Boey W, et al. Cost-utility analysis of recombinant factor VIIa (Novoseven®) in six children with long-standing inhibitors to factor VII or IX. Haemophilia 2001; 7 (3): 279–85

    Article  PubMed  CAS  Google Scholar 

Download references

Acknowledgements

The authors thank Anne Costa and Dominique Manac’h for their support. The authors have no conflicts of interest to declare. The study was partially funded by the Direction des finances Assistance Publique Hôpitaux de Paris (APHP).

Author information

Authors and Affiliations

Authors

Corresponding author

Correspondence to Nathalie Pelletier-Fleury.

Rights and permissions

Reprints and permissions

About this article

Cite this article

Galanaud, J.P., Pelletier-Fleury, N., Logerot-Lebrun, H. et al. Determinants of Drug Costs in Hospitalised Patients with Haemophilia. Pharmacoeconomics 21, 699–707 (2003). https://doi.org/10.2165/00019053-200321100-00002

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.2165/00019053-200321100-00002

Keywords

Navigation